News

BridgeBio Pharma Announces Positive Feedback from the U.S. FDA and EU EMA on the Regulatory Path for a Pivotal Phase 3 Trial of Infigratinib in Children with Achondroplasia

The following press release was obtained from BridgeBio.com, parent company of QED therapeutics. To learn more about QED and its research surrounding achondroplasia and other skeletal dysplasias, click here. –…

BioMarin · Pharmachon · Voxzogo

PHARMACHON: Pharmacokinetics of VOXZOGO

The following, written by Victoria Garcia, RN, exclusively for The Chandler Project and members of the achondroplasia community are questions and answers compiled from ‘One Family’s Story: A Guide to…

Hypochondroplasia · Infigratinib · Propel · QED

BridgeBio Pharma Presents Updated Six Month Results from its Phase 2 Cohort 5 Trial of Infigratinib in Achondroplasia at the Endocrine Society (ENDO) 2023 Annual Conference

The following press release was obtained from BridgeBio.com, parent company of QED therapeutics. To learn more about QED and their research surrounding achondroplasia and other skeletal dysplasias, click here. – Treatment with infigratinib at…

Hypochondroplasia · Infigratinib · Propel · QED

BridgeBio Pharma to Present Updated Six Month Results from its Phase 2 Cohort 5 Study of Infigratinib in Achondroplasia in a Late Breaker Oral Presentation at the Endocrine Society (ENDO) 2023 Annual Conference

Achondroplasia · Treatment · Voxzogo

Children with achondroplasia to benefit from life-changing treatment added to PBS

The Murdoch Children’s Research Institute has welcomed the news that children born with the most common form of dwarfism, achondroplasia, will have access to a new life-changing treatment on the Pharmaceutical Benefits Scheme (PBS).

Voxzogo

New Drug Treats Dwarfism

Life-changing breakthrough for children Murdoch Children’s Research Institute (MCRI) recently shared a new article from the Herald Sun titled ‘New Drug Treats Dwarfism’ written by Sue Dunlevy. In the post,…